Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System
Model scores of the 29,894 trials active now.
Model score 13% for any early termination, 1% for termination due to slow enrollment. Past trials like this one (phase, sponsor type and disease area, started 2008–2016) ended terminated 15% of the time (734 trials).
Finished trial that started in 2015 or later: the model never saw it during training.
How it ended
Terminated on 2021-11-02, after 2018-06-18 start.
Reason given by the sponsor: “safety”
The score above never used this outcome: finished trial that started in 2015 or later: the model never saw it during training.
Termination over time, as the model saw it at the start
Cumulative chance of ending terminated (red) or completed (green) by each year from its start; it actually ended on 2021-11-02. The rest of the way to 100% is still running.
From the competing-risks survival model (termination vs completion, still-running trials censored), fit to trials that started 2008–2014 and recalibrated to termination rates over 2022–2024, since termination has become more common. Checked on past years, it still ran a little low when the rise continued.
What moved the score
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Accepts healthy volunteers: No (raises risk) · Who can join
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Wording of the registration (title, summary, criteria) (raises risk) · Wording
Phrases that raised it: first dosecomply withneurologicconditiontime of
Phrases that lowered it: participantsprotocol definedopen labelmonthtreatment -
Sponsor's past termination rate: 0% (lowers risk) · Sponsor's track record
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Phase: 3 (lowers risk) · Design
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Has a data monitoring committee: not given (lowers risk) · Design
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Disease area, neurology: Yes (raises risk) · Disease area
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Trials the sponsor started in the prior 2 years: 0 (raises risk) · Sponsor's track record
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Design: single group (raises risk) · Design
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Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
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Disease area, cancer: No (lowers risk) · Disease area
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Primary purpose: treatment (raises risk) · Design
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Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
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Sponsor type: industry (lowers risk) · Sponsor's track record
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Number of arms: 1 (raises risk) · Design
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Maximum age: not given (raises risk) · Who can join
Every input that moved this score, largest first: SHAP contributions in log-odds, red raising risk and green lowering it. They describe the model, not causes of termination. Phrases are traced from the text features back to this registration's words, approximately.
The trial
Design
- Phase
- 3 (typical: 2)
- Number of arms
- 1 (typical: 2)
- Allocation
- not applicable (typical: randomized)
- Design
- single group (typical: parallel)
- Primary purpose
- treatment
- Blinding
- none
- Has a data monitoring committee
- not given (typical: 56% yes)
- Responsible party
- sponsor
- Collaborators
- 0
- Keywords listed
- 3 (typical: 1)
Who can join
- Minimum age
- 4 (typical: 18)
- Maximum age
- not given (typical: 71)
- Sex
- all
- Accepts healthy volunteers
- No (typical: 9% yes)
Sponsor's track record
- Sponsor type
- industry (typical: academic or other)
- Sponsor's past trials
- 1 (typical: 32)
- Sponsor's past termination rate
- 0% (typical: 9.9%)
- Trials the sponsor started in the prior 2 years
- 0 (typical: 19)
Drug's FDA history
- Drugs with FDA adverse-event history
- 0 (typical: 1)
- Drug has FAERS history
- No (typical: 64% yes)
- Drug's FAERS reports before start
- 0 (typical: 2148)
- Drug's FAERS reports in the prior 12 months
- 0 (typical: 412.5)
- Drug's FAERS reports marked serious
- no FAERS history (typical: 85.6%)
- Drug's FAERS reports with a death
- no FAERS history (typical: 15.9%)
Disease area and drugs
- Disease areas
- neurology, metabolic, hematology
- Drugs matched to FDA substances
- adrabetadex
Typical values are among the trials active now. Sponsor and drug history are counted only up to this trial's start date.
Similar finished trials
- Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease NCT04958642 · started 2015 · scored riskier than 21% of active trials terminated
- VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease NCT02534844 · started 2015 · scored riskier than 67% of active trials completed
- Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C NCT02612129 · started 2016 · scored riskier than 30% of active trials completed
- Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1 NCT03879655 · started 2019 · scored riskier than 53% of active trials terminated
- N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) NCT03759639 · started 2019 · scored riskier than 20% of active trials completed
Closest by meaning of the registration text (PubMedBERT embeddings), among finished trials. How they ended is context, not an input to this trial's score.